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Editorial (Dr SS Agarwal, Dr K K Aggarwal)
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15th December 2016
Rare diseases and rare drugs
  1. "Spinal Muscular Atrophy (SMA)" is a rare genetic disease. 
  2. In India, there are families whose children are suffering from this progressive disease.
  3. Due to SMA, children cannot walk, and, the nightmare does not end here as it is a progressive degenerative disease which means the kids may potentially see deterioration in function of their other limbs - hands, spines and even lungs. 
  4. Limited data suggest that survival has increased in patients with SMA type 1 born from 1995 through 2006 compared with those born from 1980 to 1994 
  5. Ventilation for >16 hours a day, use of mechanical insufflation-exsufflation device, and gastrostomy tube feeding were significantly and independently associated with prolonged survival, while year of birth was not. Thus, longer survival in the later time period appears to be related to more aggressive care.
  6. Treatments that enhance the level of SMN protein may be available in the future 
  7. Gene therapy using an adeno-associated virus vector to augment spinal cord SMN expression has shown promise in a mouse model of SMA
  8. Another promising approach involves intracerebroventricular or systemic injection of antisense oligonucleotides that effectively restore SMN expression
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Press Release
TB infection is different from TB disease?

New Delhi, 14th December, 2016: In most people who breathe in TB bacteria, the body's immune system is able to fight the TB bacteria and stop them from multiplying. This is called TB infection. People who are infected with TB do not feel sick, do not have any symptoms and cannot spread TB.
But on the other hand if the infected person's immune system cannot stop the bacteria from multiplying, the bacteria eventually cause symptoms of active TB, which is called TB disease.
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